Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (NCT NCT05267106)

Last Updated: 2025-12-30

Results Overview

ORR was defined as the percentage of participants who achieved a best overall response of complete response (CR) or partial response (PR) based on Response Assessment in Neuro-Oncology (RANO) as determined by an independent centralized radiological review committee. CR requires all of the following: disappearance of all enhancing measurable/nonmeasurable disease sustained for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires all of the following: ≥50% decrease, compared with baseline, in the sum of products of perpendicular diameters of all measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with baseline scan; on a corticosteroid dose not greater than the dose at time of baseline scan and stable/improved clinically.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

83 participants

Primary outcome timeframe

up to 651 days

Results posted on

2025-12-30

Participant Flow

This study was conducted at 31 study centers in Denmark, France, Germany, Italy, Japan, Netherlands, Spain, the United Kingdom, and the United States.

Participant milestones

Participant milestones
Measure	Recurrent Glioblastoma Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Overall Study STARTED	74	9
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	74	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Recurrent Glioblastoma Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Overall Study Death	50	2
Overall Study Study terminated by Sponsor	24	6
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Total n=83 Participants Total of all reporting groups
Age, Continuous	39.2 years STANDARD_DEVIATION 12.75 • n=166 Participants	57.0 years STANDARD_DEVIATION 11.45 • n=174 Participants	55.1 years STANDARD_DEVIATION 12.79 • n=167 Participants
Sex: Female, Male Female	3 Participants n=166 Participants	30 Participants n=174 Participants	33 Participants n=167 Participants
Sex: Female, Male Male	6 Participants n=166 Participants	44 Participants n=174 Participants	50 Participants n=167 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=166 Participants	1 Participants n=174 Participants	1 Participants n=167 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=166 Participants	60 Participants n=174 Participants	66 Participants n=167 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=166 Participants	13 Participants n=174 Participants	16 Participants n=167 Participants
Race/Ethnicity, Customized White/Caucasian	6 Participants n=166 Participants	58 Participants n=174 Participants	64 Participants n=167 Participants
Race/Ethnicity, Customized Black/African-American	0 Participants n=166 Participants	2 Participants n=174 Participants	2 Participants n=167 Participants
Race/Ethnicity, Customized Asian	0 Participants n=166 Participants	2 Participants n=174 Participants	2 Participants n=167 Participants
Race/Ethnicity, Customized Not Reported	1 Participants n=166 Participants	7 Participants n=174 Participants	8 Participants n=167 Participants
Race/Ethnicity, Customized Unknown	1 Participants n=166 Participants	5 Participants n=174 Participants	6 Participants n=167 Participants
Race/Ethnicity, Customized Captured as "Other" in Database	1 Participants n=166 Participants	0 Participants n=174 Participants	1 Participants n=167 Participants

PRIMARY outcome

Timeframe: up to 651 days

Population: Full Analysis Set: all enrolled participants who received at least 1 dose of pemigatinib. The 95% confidence interval (CI) was calculated based on the exact method for binomial distribution.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Objective Response Rate (ORR) in Participants With Recurrent Glioblastoma Based on Independent Central Review Confirmed tumor responses	5.4 percentage of participants Interval 1.49 to 13.27	—
Objective Response Rate (ORR) in Participants With Recurrent Glioblastoma Based on Independent Central Review Unconfirmed tumor responses	8.1 percentage of participants Interval 3.03 to 16.82	—

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. The 95% CI was calculated based on the exact method for binomial distribution.

ORR was defined as the percentage of participants who achieved a best overall response of CR or PR based on RANO as determined by an independent centralized radiological review committee. CR requires all of the following: disappearance of all enhancing measurable/nonmeasurable disease sustained for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires all of the following: ≥50% decrease, compared with baseline, in the sum of products of perpendicular diameters of all measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with baseline scan; on a corticosteroid dose not greater than the dose at time of baseline scan and stable/improved clinically.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
ORR in Participants With Recurrent Non-glioblastoma Central Nervous System Tumors Based on Independent Central Review Confirmed tumor responses	—	22.2 percentage of participants Interval 2.81 to 60.01
ORR in Participants With Recurrent Non-glioblastoma Central Nervous System Tumors Based on Independent Central Review Unconfirmed tumor responses	—	22.2 percentage of participants Interval 2.81 to 60.01

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. Only those participants with a confirmed CR or PR were analyzed. The 95% CIs were calculated using the Brookmeyer and Crowley method.

Duration of response was defined as the time from the first assessment of confirmed CR or PR until progressive disease (PD) (according to RANO and assessed by an independent centralized radiological review committee) or death (whichever occurred first). Progression was defined by any of the following: ≥25% increase in the sum of the products of perpendicular diameters of enhancing lesions (compared with baseline if no decrease) on stable or increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor, or to decrease in corticosteroid dose.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=4 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=2 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Duration of Confirmed Response Based on Independent Central Review	NA months The median and the lower and upper bounds of the confidence interval were not estimable because no responders had disease progression or died.	NA months The median and the lower and upper bounds of the confidence interval were not estimable because no responders had disease progression or died.

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. Only those participants with an unconfirmed CR or PR were analyzed. The 95% CIs were calculated using the Brookmeyer and Crowley method.

Duration of response was defined as the time from the first assessment of unconfirmed CR or PR until progressive disease (PD) (according to RANO and assessed by an independent centralized radiological review committee) or death (whichever occurred first). Progression was defined by any of the following: ≥25% increase in the sum of the products of perpendicular diameters of enhancing lesions (compared with baseline if no decrease) on stable or increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor, or to decrease in corticosteroid dose.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=6 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=2 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Duration of Unconfirmed Response Based on Independent Central Review	NA months Interval 1.74 to The median and the upper bound of the confidence interval were not estimable because too few responders had disease progression or died.	NA months The median and the lower and upper bounds of the confidence interval were not estimable because no responders had disease progression or died.

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. The 95% CIs were calculated based on the exact method for binomial distribution.

ORR was defined as the percentage of participants who achieved an unconfirmed best overall response of CR or PR based on RANO as determined by investigator assessment. CR requires all of the following: disappearance of all enhancing measurable/nonmeasurable disease sustained for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires all of the following: ≥50% decrease, compared with baseline, in the sum of products of perpendicular diameters of all measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with baseline scan; on a corticosteroid dose not greater than the dose at time of baseline scan and stable/improved clinically.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
ORR as Determined by Investigator Assessment	8.1 percentage of participants Interval 3.03 to 16.82	22.2 percentage of participants Interval 2.81 to 60.01

SECONDARY outcome

Timeframe: up to 814 days

Population: Safety Population: all enrolled participants who received at least 1 dose of pemigatinib

An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	73 Participants	9 Participants

SECONDARY outcome

Timeframe: up to 814 days

Population: Safety Population

An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Number of Participants With Any ≥Grade 3 TEAE	27 Participants	3 Participants

SECONDARY outcome

Timeframe: up to 814 days

Population: Safety Population

An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Number of Participants With TEAEs Leading to Discontinuation of Pemigatinib, Pemigatinib Dose Interruption, and Pemigatinib Dose Reduction TEAEs leading to discontinuation of pemigatinib	2 Participants	0 Participants
Number of Participants With TEAEs Leading to Discontinuation of Pemigatinib, Pemigatinib Dose Interruption, and Pemigatinib Dose Reduction TEAEs leading to pemigatinib dose interruption	22 Participants	4 Participants
Number of Participants With TEAEs Leading to Discontinuation of Pemigatinib, Pemigatinib Dose Interruption, and Pemigatinib Dose Reduction TEAEs leading to pemigatinib dose reduction	5 Participants	2 Participants

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. The 95% CIs were calculated based on the exact method for binomial distribution.

DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or stable disease (SD) based on RANO as determined by an independent centralized radiological review committee. In the case of SD, measurements must have met the SD criteria after the date of the first dose at a minimum interval of 42 days. SD occurred if the participant didn't qualify for CR, PR, or PD and required the following: stable nonenhancing (T2/FLAIR) lesions on same or lower dose of corticosteroids compared with baseline scan and clinically stable status.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Disease Control Rate (DCR) Based on Independent Central Review Confirmed tumor responses	36.5 percentage of participants Interval 25.6 to 48.49	66.7 percentage of participants Interval 29.93 to 92.51
Disease Control Rate (DCR) Based on Independent Central Review Unconfirmed tumor responses	36.5 percentage of participants Interval 25.6 to 48.49	66.7 percentage of participants Interval 29.93 to 92.51

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. The 95% CIs were calculated using the Brookmeyer and Crowley method.

PFS was defined as the time from the first dose until progressive disease (according to RANO and assessed by an independent centralized radiological review committee) or death (whichever occurred first). Progression was defined by any of the following: ≥25% increase in the sum of the products of perpendicular diameters of enhancing lesions (compared with baseline if no decrease) on stable or increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor, or to decrease in corticosteroid dose.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Progression-free Survival (PFS) Based on Independent Central Review	2.07 months Interval 2.04 to 2.37	NA months Interval 1.74 to The median and the upper bound of the confidence interval were not estimable because too few participants had disease progression or died.

SECONDARY outcome

Timeframe: up to 784 days

Population: Full Analysis Set. The 95% CIs were calculated using the Brookmeyer and Crowley method.

Overall survival was defined as the time from the first dose of study drug to death due to any cause.

Outcome measures

Outcome measures
Measure	Recurrent Glioblastoma n=74 Participants Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 Participants Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
Overall Survival	11.37 months Interval 9.23 to 14.85	24.08 months Interval 18.79 to The upper bound of the confidence interval was not estimable because too few participants died.

Adverse Events

Recurrent Glioblastoma

Serious events: 17 serious events

Other events: 71 other events

Deaths: 50 deaths

Recurrent Non-glioblastoma CNS Tumors

Serious events: 0 serious events

Other events: 9 other events

Deaths: 2 deaths

Total

Serious events: 17 serious events

Other events: 80 other events

Deaths: 52 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Incyte Corporation

Phone: 1-855-463-3463

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Publication restrictions are in place

Restriction type: OTHER

Measure	Recurrent Glioblastoma n=74 participants at risk Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Recurrent Non-glioblastoma CNS Tumors n=9 participants at risk Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.	Total n=83 participants at risk Total
Renal and urinary disorders Acute kidney injury	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Aphasia	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
General disorders Asthenia	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Cognitive disorder	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Psychiatric disorders Confusional state	2.7% 2/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	2.4% 2/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Epilepsy	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Gastrointestinal disorders Faecaloma	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Injury, poisoning and procedural complications Fall	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Haemorrhage intracranial	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Hemiparesis	2.7% 2/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	2.4% 2/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Infections and infestations Herpes zoster	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Gastrointestinal disorders Intestinal perforation	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Injury, poisoning and procedural complications Lumbar vertebral fracture	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Partial seizures with secondary generalisation	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Infections and infestations Pneumonia	2.7% 2/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	2.4% 2/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Nervous system disorders Seizure	2.7% 2/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	2.4% 2/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Infections and infestations Sepsis	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Injury, poisoning and procedural complications Stroke-like migraine attacks after radiation therapy	1.4% 1/74 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 1 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Infections and infestations Urinary tract infection	2.7% 2/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	2.4% 2/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
Injury, poisoning and procedural complications Wound dehiscence	1.4% 1/74 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	0.00% 0/9 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.	1.2% 1/83 • Number of events 2 • up to 814 days Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.